12/03/2018
Dr Annie Huang, Principal Investigator at The Arthur and Sonia Labatt Brain Tumour Research Centre Canada, shares how her team are using samples collected by The Children’s Hospital at Westmead Tumour Bank to discover better treatments for low-prognosis cancer in children.

Atypical teratoid rhabdoid tumours (ATRTs) are rare tumours that attack the brain and historically have been considered incurable. ATRTs are among the most common malignant brain tumours in children less than three years of age. Patients are typically treated with a combination of chemotherapy and radiation, and survival rates are low.
 
Up until now, the medical community has generally considered ATRTs as a single disease as there is one gene that is lost in all the tumours – typically a combination of drugs are used to treat ATRTs, but there is no well-proven or established method of treatment.
 
Because ATRTs have been known to be so aggressive and rare, they haven’t received much attention.
 
The Rare Brain Tumor Consortium was created to enable researchers like us to collect large numbers of these tumours. For one study alone (Integrated (epi)-genomic analyses identify subgroup-specific therapeutic targets to CNS rhabdoid tumours), we collaborated with colleagues from close to 40 countries around the world, including The Tumour Bank at The Children’s Hospital at Westmead which has received vital funding from The Kids’ Cancer Project for well over a decade.
 
Tumour banks played an important role allow us to amass over 200 of these rare tumours, which we only see two to four times a year at SickKids Hospital in Toronto, Canada.
 
The Tumour Bank at The Children’s Hospital at Westmead has been instrumental in helping us with our ATRTs study along with other projects including studies on ETMRs, pineoblastoma and other rare embryonal tumours.
 
During our investigation, we have confirmed our clinical observation that ATRTs are not uniform – there are three separate epigenetic subtypes, each associated with different clinical and patient profiles. That is, each of these tumour types come from different cells that form different parts of the brain. One type in particular – what we called group 2 ATRTs in the study – are particularly resistant to many different therapies, but we discovered that they respond favourably to two well-known cancer drugs, Dasatinib and Nilotinib. Because Dasatinib and Nilotinib have been used widely in leukaemia treatment, we know they are generally well tolerated in children, so we’re excited to incorporate these drugs into an ATRT trial.
 
 

The long-term goal of research into childhood malignancies is to reduce the incidence of cancer and to improve the outlook of children suffering from this disease. It is through research that we will gain the knowledge about cancer that will eventually lead to new approaches in therapy. However, such research is dependent upon the availability of cancer specimens for the scientist to study.
 
We now know ATRT is not an incurable disease – we just have to figure out creative ways to treat it.


Author: Dr Annie Huang, The Hospital for Sick Children, and Principal Investigator at The Arthur and Sonia Labatt Brain Tumour Research Centre Toronto, Canada.
 
This article was originally published in The Children’s Hospital of Westmead Tumour Bank Newsletter, December 2017 Edition.
Images kindly supplied by Annie Huang.